Key Highlights of SpringWorks Therapeutics - OGSIVEO (nirogacestat) is the first and only FDA-approved therapy for desmoid tumors[15] - GOMEKLI (mirdametinib) is the first and only FDA-approved therapy for both adults and children with NF1-PN[15] - SpringWorks Therapeutics expects profitability in the first half of 2026[10] - SpringWorks Therapeutics had $461.9 million in cash, cash equivalents, and marketable securities[132] OGSIVEO (Nirogacestat) for Desmoid Tumors - OGSIVEO generated $61.5 million in net revenue in Q4 2024 and $172.0 million in FY 2024[15] - Between October 2023 and October 2024, approximately 11,000 patients with desmoid tumor ICD-10 claims were identified[15] - Objective response rate (ORR) increased from 34.3% with up to 1 year of treatment to 45.7% with up to 4 years of treatment[48] GOMEKLI (Mirdametinib) for NF1-PN - GOMEKLI is approved for the treatment of adult and pediatric patients 2 years of age and older with NF1-PN[10] - The U S market includes approximately 30,000 adult and 10,000 pediatric patients with NF1-PN[15] - In clinical trials, GOMEKLI showed a confirmed overall response rate of 41% in adults (n=58) and 52% in pediatrics (n=56)[67]

Core Insights - BrightSpring Health Services' specialty pharmacy, Onco360®, has been selected as the national pharmacy partner for newly approved therapies targeting advanced cancers and rare genetic disorders [1][3]. Company Overview - BrightSpring Health Services offers integrated home- and community-based pharmacy and health solutions for complex populations, serving over 400,000 customers daily across all 50 states [4]. - Onco360® is a leading independent oncology pharmacy founded in 2003, focusing on the specialized needs of oncologists, patients, and healthcare providers [5]. New Therapy Partnerships - Onco360® will provide access, education, data, and expert support for innovative treatments for advanced ovarian and lung cancers, as well as neurofibromatosis type 1 [2][3]. - The therapies include GOMEKLI™ for neurofibromatosis type 1, AVMAPKI™ FAKZYNJA™ CO-PACK for KRAS-mutated recurrent low-grade serous ovarian cancer, and IBTROZI™ for ROS1-positive non-small cell lung cancer [6].

Core Viewpoint - SpringWorks Therapeutics has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use for its MEK inhibitor, mirdametinib, aimed at treating a rare genetic disorder [1][2]. Company Developments - The CHMP has recommended conditional marketing authorization for mirdametinib to treat symptomatic, inoperable plexiform neurofibromas in patients aged two years and older with neurofibromatosis type 1 [2]. - If approved, mirdametinib will be the first therapy authorized for treating NF1-PN in the European Union [3]. - The FDA approved mirdametinib under the brand name Gomekli in February 2025, generating sales of $4.9 million in the U.S. during Q1 2025 [3]. Financial Performance - Year-to-date, SpringWorks shares have increased by 28.6%, contrasting with a 5.5% decline in the industry [5]. - Merck KGaA announced plans to acquire SpringWorks for $47 per share, totaling approximately $3.9 billion, with the transaction expected to close in the second half of 2025 [6]. Product Pipeline - Ogsiveo, an oral gamma-secretase inhibitor, was approved in the U.S. for treating desmoid tumors in November 2023 and generated $172 million in sales in 2024, with $44.1 million in Q1 2025 [7][8]. - Ongoing label expansion studies for Ogsiveo are targeting various cancer indications, with the EMA's opinion on its MAA expected in Q2 2025 [8]. Market Position - The acquisition by Merck KGaA is anticipated to enhance SpringWorks' ability to reach global markets and support patients with rare tumors [9].

证券研究报告 | 行业周报 2025 年 05 月 06 日 医药 本周医药板块上涨 0.49%，百济神州 BCL-2 抑制剂申报上市 | 股票 | 股票 | 投资 | EPS（元） | | PE | | | --- | --- | --- | --- | --- | --- | --- | | 代码 | 名称 | 评级 | 2025E | 2026E | 2025E | 2026E | | 002755.SZ | 奥赛康 | 买入 | 0.2 | 0.28 | 93.95 | 67.11 | | 01952.HK | 云顶新耀 | 买入 | -0.94 | 0.58 | - | - | | 600079.SH | 人福医药 | 增持 | 1.59 | 1.86 | 13.13 | 11.22 | | 688192.SH | 迪哲医药 | 增持 | -1.33 | -0.08 | -43.54 | -723.88 | | 688315.SH | 诺禾致源 | 买入 | 0.53 | 0.62 | 26.53 | 22.68 | | 688321.SH | 微芯生物 | 增持 | 0.05 | 0.23 | 3 ...

每经记者 甄素静 每经编辑 张益铭 丨2025年4月29日 星期二丨 NO.1 药明康德一季度净利润同比增长89.06% 4月28日，药明康德公告称，2025年第一季度实现营业收入96.55亿元，同比增长20.96%；归属于上市公 司股东的净利润36.72亿元，同比增长89.06%。 点评：药明康德2025年一季度营收、净利双增，展现强劲增长势头，凸显其在医药研发服务领域强大竞 争力与行业领先地位。 NO.2 派格生物通过港交所聆讯即将上市，8轮融资估值40亿 派格生物近日通过聆讯，拟在香港主板上市，独家保荐人为中金。公司是自主研究及开发慢性疾病创新 疗法的生物技术公司。2024年收入0.07亿元，净亏损2.83亿元。 点评：派格生物通过港交所聆讯，即将登陆港股。其专注慢性疾病创新疗法，虽目前亏损，但核心产品 潜力大，8轮融资获40亿估值，上市后有望借资本加速发展。 NO.3 德国默克将以39亿美元收购SpringWorks 4月28日，德国默克宣布，将收购SpringWorks，交易代价约为39亿美元的股权价值，或根据 SpringWorks截至2024年12月31日的现金余额计算的34亿美元企业价值。 点 ...

Core Viewpoint - SpringWorks Therapeutics, Inc. anticipates a favorable opinion from the CHMP of the EMA regarding the marketing authorization application for nirogacestat in Q2 2025, aimed at treating adults with desmoid tumors [1] Company Overview - SpringWorks Therapeutics is a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, with FDA-approved products including OGSIVEO (nirogacestat) for desmoid tumors and GOMEKLI™ (mirdametinib) for neurofibromatosis type 1 associated plexiform neurofibromas [2]